JAG101

JAG101 is an investigational AAV9-based gene replacement therapy being developed by Jaguar Gene Therapy for the treatment of Type 1 galactosemia, a rare autosomal recessive metabolic disorder caused by loss-of-function mutations in the galactose-1-phosphate uridylyltransferase (GALT) gene that lead to toxic accumulation of galactose, galactose-1-phosphate (Gal-1P), and galactitol.[1][6][8][9][10][16] JAG101 is designed as a one-time intravenous therapy that delivers a functional GALT gene via an AAV9 vector to restore GALT enzyme expression and activity in key tissues such as liver, brain, and skeletal muscle, with the goal of rapidly and durably reducing pathogenic metabolites and preventing or mitigating acute neonatal decompensation, cataracts, and long-term cognitive, neurological, and reproductive complications.[1][4][6][8][9][15][16] Preclinical studies in GALT-deficient mouse and rat models have shown dose-dependent increases in GALT expression and activity and significant reductions in galactose, Gal-1P, and galactitol in plasma, liver, brain, and muscle, along with decreased cataract incidence, supporting further development of JAG101 as a disease-modifying therapy for Type 1 galactosemia.[1][4][6][8][15][16]